Form 8-K

 

 

UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

Washington, D.C. 20549

 

 

FORM 8-K

 

 

CURRENT REPORT

PURSUANT TO SECTION 13 OR 15(d) OF

THE SECURITIES EXCHANGE ACT OF 1934

Date of report (Date of earliest event reported): March 13, 2013

 

 

ZIOPHARM Oncology, Inc.

(Exact Name of Registrant as Specified in Charter)

 

 

 

Delaware   001-33038   84-1475672

(State or Other Jurisdiction

of Incorporation)

 

(Commission

File Number)

 

(IRS Employer

Identification No.)

1180 Avenue of the Americas

20th Floor

New York, NY

  10036
(Address of Principal Executive Offices)   (Zip Code)

(646) 214-0700

(Registrant’s telephone number, including area code)

Not applicable

(Former Name or Former Address, if Changed Since Last Report)

 

 

Check the appropriate box below if the Form 8-K is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:

 

¨ Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425).

 

¨ Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12).

 

¨ Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b)).

 

¨ Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c)).

 

 

 


Item 8.01 Other Events

ZIOPHARM Oncology, Inc., or the Company, will present the attached discussion of the Company’s palifosfamide development strategy and milestones, as well as the Company’s synthetic biology program, at the Barclays Global Healthcare Conference in Miami, Florida, being held on March 13, 2013.

A copy of the above referenced presentation is filed as Exhibit 99.1 to this Current Report on Form 8-K.

 

Item 9.01 Financial Statements and Exhibits

 

  (d) Exhibits

 

Exhibit

No.

 

Description

99.1   Presentation of the Company dated March 13, 2013

 

2


SIGNATURES

Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.

 

    ZIOPHARM Oncology, Inc.
    By:  

/s/ Jason A Amello

Date: March 13, 2013     Name:   Jason A. Amello
    Title:  

Executive Vice President, Chief Financial

Officer and Treasurer

 

3


INDEX OF EXHIBITS

 

Exhibit

No.

 

Description

99.1   Presentation of the Company dated March 13, 2013

 

4

EX-99.1
Better Cancer Medicine
Barclays Global Healthcare Conference
Jonathan Lewis, MD, PhD
Chief Executive Officer
Exhibit 99.1
1
March 13, 2013


Forward-Looking Statements
This presentation contains certain forward-looking statements about ZIOPHARM Oncology that are intended to be covered by the safe
harbor for “forward-looking statements”
provided by the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking
statements are statements that are not historical facts. Words such as “expect(s),”
“feel(s),”
“believe(s),”
“will,”
“may,”
“anticipate(s)”
and
similar expressions are intended to identify forward-looking statements. These statements include, but are not limited to, statements
regarding our ability to successfully develop and commercialize our therapeutic products; our ability to expand our long-term business
opportunities; our expectations regarding approval for our products by the U.S. Food and Drug Administration or equivalent foreign
regulatory agencies; estimates regarding the market potential for our products; financial projections and estimates and their underlying
assumptions; and future performance. All of such statements are subject to certain risks and uncertainties, many of which are difficult to
predict and generally beyond the control of the Company, that could cause actual results to differ materially from those expressed in, or
implied or projected by, the forward-looking statements. These risks and uncertainties include, but are not limited to: whether any of our
therapeutic candidates will advance further in the clinical trials process and whether and when, if at all, they will receive final approval
from the U.S. Food and Drug Administration or equivalent foreign
regulatory agencies and for which indications; whether any of our
therapeutic candidates will be successfully marketed if approved; whether our therapeutic product discovery and development efforts will
be successful; our ability to achieve the results contemplated by our collaboration agreements; the strength and enforceability of our
intellectual property rights; competition from pharmaceutical and biotechnology companies; the development of and our ability to
take
advantage of the market for our therapeutic products; our ability to raise additional capital to fund our operations on terms acceptable to
us; general economic conditions; and the other risk factors contained in our periodic and interim reports filed with the SEC including, but
not limited
to,
our
Annual
Report
on
Form
10-K
for
the
fiscal
year
ended
December
31,
2011,
and
our
Quarterly
Report
on
Form
10-Q for
the fiscal quarter ended September 30, 2012. Our audience is cautioned not to place undue reliance on these forward-looking statements
that speak only as of the date hereof, and we do not undertake any obligation to revise and disseminate forward-looking statements to
reflect events or circumstances after the date hereof, or to reflect the occurrence of or non-occurrence of any events.
2
March 13, 2013


Small
Molecules
Synthetic
Biology
Better Cancer Medicine
Diverse Portfolio of Therapeutics Addressing
Unmet Medical Needs in Cancer
Paradigm Shifting Approaches
to Impact and Cure Cancer
3
March 13, 2013


Data-Driven Oncology Portfolio
4
March 13, 2013
Palifosfamide
Soft Tissue Sarcoma
Small Cell Lung Cancer
Oral Formulation
Ad-RTS-IL-12
Melanoma
Breast (+palifosfamide)
Other Candidates
Indibulin
Breast Cancer
Darinaparsin
PTCL
Compound
Preclinical
Phase 1
Phase 2
Phase 3


Palifosfamide
Two Phase 3 programs
Metastatic soft tissue sarcoma
Small cell lung cancer
Potential in multiple tumor types
Exceptional intellectual property profile (t0 2029)
Synthetic Biology
Two Phase 2 programs
Melanoma
Breast cancer
Exploring multiple opportunities for new-paradigm treatment modality
Controlled switch for in vivo protein production
Near-Term Value Drivers
5
March 13, 2013


Major Clinical Milestones Over Next 18 Months
Q1-13
Q2-13
Q3-13
Q4-13
Q1-14
Q2-14
Possible NDA Submission
TOP LINE DATA
Possible ASCO Late-breaking
Interim Analysis
Palifosfamide
IL-12 DNA
Possible Phase 1 Melanoma ASCO Presentation
6
March 13, 2013
MATISSE
PHASE 2 MELANOMA
PHASE 2 BREAST CANCER (COMBO)
PICASSO
3


Palifosfamide
7
March 13, 2013


Broad Application
Effect in solid tumors and hematological malignancies
Safety, Efficacy and
Accessibility
Potent bi-functional DNA alkylating agent that has
activity in multiple tumors by evading typical
resistance pathways (in class with bendamustine,
cyclophosphamide, and ifosfamide), with low toxicity
and ease of administration
Addressing Unmet Needs
Orphan Drug status established for STS in U.S. and
Europe
Exceptional Intellectual
Property Profile
U.S. pharmaceutical composition patent rights
extending to 2029; other pending applications WW
A Potent Bi-Functional DNA Alkylating Agent
8
March 13, 2013


Soft Tissue Sarcoma
High unmet medical need; no first-line drug approved in ~30 years
First-line metastatic, non-GIST STS:
1
Approximately 9,000 in U.S.
2
Approximately 14,000 in Europe
Market in transformation
9
March 13, 2013
April 2012 FDA full approval of Votrient
®
for second-line
therapy (PFS endpoint); August 2012 European full approval
1
Source: U.S.:  IntrinsiQ Data, © Copyright 2012, IntrinsiQ, LLC an AmerisourceBergen Specialty Group company.  All rights reserved; 
remainder of world Company estimated from epidemiology (SEER, NCCN).
2
IntrinsiQ: # new patient prescriptions written for first-line, non-GIST STS over 12 months.


1
10
1
Votrient
®
ODAC
March
20,
2012


Phase 3 Design (PICASSO 3)
N:
447 subjects; first-line metastatic STS
Regimen:
Palifosfamide + doxorubicin vs. placebo + doxorubicin
Primary Endpoint:
PFS for full approval
Powering:
Powered for both PFS and OS
PFS Analysis:
Independent blinded radiologic review; Efficacy analysis by IDMC
IDMC following pre-determined number of PFS events
Results:
Pivotal topline PFS data, OS futility analysis (OS remains blinded)
to be announced week of March 25, 2013
11
March 13, 2013


PFS primary endpoint in Phase 3 registration trials and
now supportive for certain indications:
In 2011, 4 oncology products approved based on PFS as primary
trial endpoint
In 2012, 7 oncology products approved based on PFS as primary
trial
endpoint,
including
pazopanib
(Votrient
®
)
receiving
full
approval for STS
Regulatory Strategy: Evolving Landscape of
PFS as Primary Endpoint
12
March 13, 2013


Multicenter, Randomized, Stratified, Balanced
Phase 2 Data (PICASSO)
Source: Best of ASCO 2010 (PFS Data), internal analysis (OS Data)   
* Expected 2-yr survival is 25% based on evaluation of randomized data
** Control arm confirmed by EORTC ifosfamide vs doxorubicin study, ESMO 2012
13
March 13, 2013
Phase 3: Correctly Modeled and Powered for PFS & OS
PFS: median
7.8 mos.
4.4 mos.
PFS: hazard ratio
0.43
OS: hazard
ratio (with crossover)
0.78
2-yr survival (with crossover)*
40%
30%
Safety
Similarity between arms
No
encephalopathy, hemorrhagic cystitis, Fanconi's Syndrome
Grade 3+ events
Neutropenia and elevated creatinine (
similar between arms
)
:
Pali+Dox
:
Dox**
Arm
A
Arm
B


EORTC 62012 Randomized Phase 3 Trial (ESMO)
PICASSO
EORTC
Appropriate Risk/Benefit Ratio Is Critical
14
March 13, 2013
Pali+Dox
Dox
Ifos+Dox
Dox
PFS: median
7.8 mos.
4.4 mos.
7.4 mos.
4.6 mos.
PFS: hazard ratio
0.43
0.74
OS: hazard
ratio (with
crossover
)
0.78
2yr. N/A (mOS: 0.83)
2-yr survival
40%
30%
31%
28%
Safety
Similarity between arms
No
encephalopathy, hemorrhagic
cystitis, Fanconi's Syndrome
Dropouts for toxicity
17.6% (I+D) vs.
2.6% (D) (including toxic death)
Grade 3+ events
Neutropenia and elevated creatinine
(similar between arms)
Febrile Neutropenia
45.9% (I+D) vs.
13.6% (D)


Small Cell Lung Cancer
High unmet medical need; no innovation in decades
Worldwide
market
opportunity:
250,000+patients
1
30,000-35,000 patients in the U.S.
Rationale: ifosfamide only first-line therapy to show benefit added to
SOC
(platinum
plus
etoposide)
-
excessive
toxicity
2
Palifosfamide added to SOC demonstrating early success in highly
refractory
patients
-
combination
well
tolerated
1
SEER, Globocan.
2
15
March 13, 2013
Hoosier Oncology Group - Einhorn et. al.


MATISSE Adaptive Trial Design
Single pre-planned
evaluation by the IDMC
125
Events
Continue trial with decreased sample size
Complete trial as planned
Continue trial with increased
event size
16
March 13, 2013
Optimize chances of success
Stop trial for efficacy/futility
Complete trial as planned or resize/
repower study


MATISSE
trial
enrollment
ahead
of
projection
Successful IDMC safety review of initial 20 patients
Interim analysis of 125 events expected end of 2013
MATISSE Trial Progress
17
March 13, 2013


Recent,
U.S.
orphan
drug
launches
in
oncology
priced
at
$9K
-
$23K
per cycle range
Examples
of
products:
Adcetris
,
Caprelsa
®
,
Zelboraf
®
Market Potential
Scenarios
of
U.S.
Only
Peak
Gross
Revenues
in
$Million
Palifosfamide Price Per Patient Per Year
Patients
Treated
$ 54,000
$ 72,000
$ 90,000
Soft Tissue
Sarcoma
4,000
$ 216
$ 288
$ 360
4,500
$ 243
$ 324
$ 405
5,000
$ 270
$ 360
$ 450
Small Cell
Lung Cancer
8,000
$ 432
$576
$ 720
10,000
$ 540
$ 720
$ 900
12,000
$ 648
$ 864
$ 1,o80
18
SCLC
March 13, 2013
>$1
Bn
total
global
market
potential
for
palifosfamide
in
STS
and


Synthetic Biology
19
March 13, 2013


Revolutionary technology for precise, controlled delivery of DNA
expressing therapeutic proteins in vivo
Lead Ad-RTS-IL-12 candidate in melanoma, breast cancer
Next generation of therapeutic approaches in research pipeline
Modular inducible cancer immunotherapy (MICI)
Antibody therapy
Multigenic approaches
Synthetic Biology
20
March 13, 2013


Using Natural Cell Biology to Produce Proteins:
Adenoviral Approach
Oral ligand activates DNA
transcription, protein
production begins
Target Cell
21
March 13, 2013
mRNA
Translation
Therapeutic
proteins
Adaptable
Controlled
Precision


Phase 1/2 Ad-RTS-IL-12 Candidate
Ad-RTS-IL-12 Phase 1b in metastatic melanoma
Biologically effective dose was determined
Clinical activity observed in 71% of patients (n=7),
two highest dose cohorts
No dose-limiting toxicity reported to date
Ad-RTS-IL-12 Phase 2 in metastatic melanoma
Multi-center, single-arm, open-label expansion stage
study
Enrolling up to 15 patients
Baseline
Necrosis prior
to cycle 2
22
March 13, 2013


Design
Randomized, open-label study of Ad-RTS-IL-12 monotherapy
or
combination with palifosfamide in patients with
recurrent/metastatic breast cancer and accessible lesions
N
Up to 68 subjects
Population
Recurrent/metastatic breast cancer
Primary Endpoint
16-week PFS rate
Select Secondary
Endpoints
Objective response rate by modified RECIST v1.1
Duration of response
Evaluate pharmacodynamic tumor markers
Outcome
Changing regulatory landscape of breast cancer
Acceptance of pCR (pathologic CR) as a validated
endpoint for accelerated approval in neoadjuvant setting
Potential rapid positioning of this combination from
refractory to neoadjuvant setting
Phase 2 Breast Cancer
Ad-RTS-IL-12 + palifosfamide
23
March 13, 2013


Using Synthetic and Natural Cell Biology to Produce Proteins:
Intramuscular Plasmid DNA
EORTC-NCI-AACR International Conference on Molecular Targets and Cancer Therapeutics 2012 (animal model).
Therapeutic
Proteins
Engineered DNA Transgene
Plasmids
Orally Activated
Sustained protein production and therapeutic anti-tumor efficacy drawing
from only a single intramuscular administration of plasmid DNA.
24
March 13, 2013


Next Generation: Multigenic Approach
IP1:
LacZ
IP1:
LacZ
rLUC
rLUC
Stuffer
Stuffer
Stuffer
Stuffer
Stuffer
Stuffer
SP-RTS
SP-RTS
IP2:
fLuc
IP2:
fLuc
rLUC
rLUC
Stuffer
Stuffer
Stuffer
Stuffer
Stuffer
Stuffer
SP-RTS
SP-RTS
IP3:
SEAP
IP3:
SEAP
rLUC
rLUC
Stuffer
Stuffer
Stuffer
Stuffer
Stuffer
Stuffer
SP-RTS
SP-RTS
IP4:
GUS
IP4:
GUS
rLUC
rLUC
Stuffer
Stuffer
Stuffer
Stuffer
Stuffer
Stuffer
SP-RTS
SP-RTS
IP4:
GUS
IP4:
GUS
rLUC
rLUC
SP-RTS
SP-RTS
IP1:
LacZ
IP1:
LacZ
IP2:
fLuc
IP2:
fLuc
IP3:
SEAP
IP3:
SEAP
0.0
1.0
2.0
3.0
4.0
5.0
6.0
7.0
8.0
LacZ/rLUC Uninduced
fLUC/rLUC Uninduced
SEAP/rLUC Uninduced
GUS/rLUC Uninduced
LacZ/rLUC Induced
fLUC/rLUC Induced
SEAP/rLUC Induced
GUS/rLUC Induced
Conclusion:
4 Inducible gene programs can
be placed in parallel on the
same vector without affecting gene program performance
#: Not significantly different than
background (p>0.05)
*: Not significantly different than
the control (p>0.05)
25
March 13, 2013


Primary shares outstanding: approximately 83.2M
Cash: approximately $95.3M @ 9/30/12
Current cash resources expected to support operations
into 2H 2013
Financial Highlights
26
March 13, 2013


Major Clinical Milestones Over Next 18 Months
Q1-13
Q2-13
Q3-13
Q4-13
Q1-14
Q2-14
Possible NDA Submission
TOP LINE DATA
Possible ASCO Late-breaking
Interim Analysis
Palifosfamide
IL-12 DNA
Possible Phase 1 Melanoma ASCO Presentation
27
March 13, 2013
PHASE 2 BREAST CANCER (COMBO)
MATISSE
PICASSO
3
PHASE 2 MELANOMA


Better Cancer Medicine
Barclays Global Healthcare Conference
Jonathan Lewis, MD, PhD
Chief Executive Officer
28
March 13, 2013